Dr. Kevin Watt and his team at the Murdoch Children’s Research Institute (MCRI) in Melbourne are pioneering a novel stem cell therapy that could revolutionize the treatment of heart failure in children. By leveraging the discovery of Dr. Shinya Yamanaka, who found that specialized cells could be reprogrammed into immature stem cells, Watt's team has developed a method to convert these stem cells into heart cells using small molecules. These heart cells are then used to create small heart organoids in the lab, which can be injected into the failing hearts of children.
The therapy has already demonstrated effectiveness in animal models, including mice, pigs, and sheep, and is on the verge of entering human clinical trials. The approach targets congenital heart failure and the adverse effects of chemotherapy, which can lead to heart failure in up to 15% of cases. The treatment aims to correct these conditions by stitching large sheets of heart tissue into the failing heart, offering a new lease on life for millions of children worldwide.
Dr. Watt emphasizes the urgency of addressing heart failure, an unmet clinical challenge that affects over 500,000 children globally. The team's research focuses on using induced pluripotent stem cells (iPSCs) to convert patients' blood or skin cells into stem cells, which are then transformed into heart cells or engineered heart tissues. This personalized medicine approach, while costly, holds the promise of significantly improving the quality of life for children with heart failure.
In addition to developing cell-based treatments, the MCRI team is also utilizing stem cells to discover new drugs for heart failure. By creating miniature heart tissues from stem cells, researchers can model diseases in a dish, identifying new drug targets and advancing the development of effective therapies. The success of this innovative research is heavily reliant on philanthropic support, which Dr. Watt acknowledges as critical to accelerating the development of these transformative treatments and making them accessible to every child in need.
